Cystic Fibrosis Clinical Trial
Official title:
Exploring Caregiver Needs While Supporting People With CF Learning About Lung Transplant
Lung transplant is an option for treating end-stage lung disease in cystic fibrosis (CF). In the United States, more people with CF and low lung function die each year than undergo lung transplant. More than half of people with CF who die without a lung transplant were never referred for consideration. Patient preference not to undergo lung transplant may account for 25-40% of decisions to defer referral. Patients' health discussion networks function to support individuals in health related matters and may provide critical support during the lung transplant journey. Increasing awareness of lung transplant, and promoting the process of deliberation and utilization of social support, could reduce the number of people with CF who die without lung transplant. Additionally, the most common patient-endorsed barrier to lung transplant discussions is a worry about being a burden on family and friends after lung transplant. For lung transplant recipients with complex post-operative courses, low social support is associated with increased mortality. Additionally, adequate social support is a requirement at all lung transplant programs in the US. Investigators are interested in understanding how caregivers may benefit from using lung transplant educational resources and how caregivers prepare for having discussions with their loved ones and/or helping them make decisions about lung transplant as a treatment option for advanced CF. The purpose of this study is to test whether an investigator-designed research website compared to no caregiver intervention reduces caregiver burden (assessed with the Brief Assessment Scale for Caregivers, BASC), caregiver preparedness for lung transplant discussions, and caregiver lung transplant knowledge as an ancillary study in a multicenter RCT. Further, investigators will assess patient perceptions of caregiver support as measured by the Social Support Effectiveness Questionnaire (SSE-Q) and evaluate caregivers' willingness to provide support through semi-structured interviews in patient-caregiver dyads. Study involvement will span 6 months and study activities will involve the following: - Three Zoom research sessions (15-90 minutes each) - Survey assessments and an interview - Access to a research website that contains educational resources about lung transplant
Status | Recruiting |
Enrollment | 132 |
Est. completion date | July 31, 2027 |
Est. primary completion date | September 30, 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Years and older |
Eligibility | Inclusion Criteria: - Identified as a caregiver or loved one of an individual with CF enrolled in the Lung Transplant READY CF 2 parent RCT, or - An individual with CF enrolled in the Lung Transplant READY CF 2 parent RCT Exclusion Criteria: - People who are unable to provide informed consent - Unable to read or understand English or Spanish to complete surveys or access the website (currently only available in English and Spanish) |
Country | Name | City | State |
---|---|---|---|
United States | University of Washington Medical Center - Montlake | Seattle | Washington |
Lead Sponsor | Collaborator |
---|---|
University of Washington | Cystic Fibrosis Foundation |
United States,
Hartzler AL, Bartlett LE, Hobler MR, Reid N, Pryor JB, Kapnadak SG, Berry DL, Lober WB, Goss CH, Ramos KJ; Take on Transplant Study Group. Take on transplant: human-centered design of a patient education tool to facilitate informed discussions about lung transplant among people with cystic fibrosis. J Am Med Inform Assoc. 2022 Dec 13;30(1):26-37. doi: 10.1093/jamia/ocac176. — View Citation
Ramos KJ, Smith PJ, McKone EF, Pilewski JM, Lucy A, Hempstead SE, Tallarico E, Faro A, Rosenbluth DB, Gray AL, Dunitz JM; CF Lung Transplant Referral Guidelines Committee. Lung transplant referral for individuals with cystic fibrosis: Cystic Fibrosis Foundation consensus guidelines. J Cyst Fibros. 2019 May;18(3):321-333. doi: 10.1016/j.jcf.2019.03.002. Epub 2019 Mar 27. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Brief Assessment Scale for Caregivers (BASC): by patient's initial randomization status (intervention or attention control in parent RCT). | Investigators will compare Brief Assessment Scale for Caregivers (BASC) for caregivers of patients initially randomized to intervention in the parent RCT (patient had a chance to learn about lung transplant before their caregiver gained access) versus those randomized to the intervention at the same time as their caregiver (attention control arm initially + caregiver in intervention) versus those whose caregivers had no access to the intervention (patient in either study arm in parent RCT initially + caregiver with no access). All patient participants in the parent RCT will have access to the intervention during the 3-months when the caregivers are randomized to intervention or control. Investigators will assess changes from baseline to 3 months (no caregivers have access to the intervention) and change from 3 to 6 months (caregivers randomized to intervention or control/no access). | Measured at the baseline, 3-month, and 6-month study visits | |
Other | Social Support Effectiveness Questionnaire (SSE-Q): by patient's initial randomization status (intervention or attention control in parent RCT). | Investigators will compare Social Support Effectiveness Questionnaire (SSE-Q) for patients initially randomized to intervention in the parent RCT (patient had a chance to learn about lung transplant before their caregiver gained access) versus those randomized to the intervention at the same time as their caregiver (attention control arm initially + caregiver in intervention) versus those whose caregivers had no access to the intervention (patient in either study arm in parent RCT initially + caregiver with no access). All patient participants in the parent RCT will have access to the intervention during the 3-months when the caregivers are randomized to intervention or control. Investigators will assess changes from baseline to 3 months (no caregivers have access to the intervention) and change from 3 to 6 months (caregivers randomized to intervention or control/no access). Higher scores on the SSE-Q indicate more effective support, with a range from 0 to 80. | Measured at the baseline, 3-month, and 6-month study visits | |
Other | Assessment of Brief Assessment Scale for Caregivers (BASC): among caregivers of members of communities of concern (low socioeconomic status, Hispanic ethnicity, or Black/Asian/Other race). | Investigators will assess Brief Assessment Scale for Caregivers (BASC) within the caregivers of patient participants who are members of communities of concern. In an intention-to-treat assessment, investigators will assess the difference in caregivers' mean change in BASC in the intervention versus control arms of the study from the 3-month to the 6-month study visit using linear mixed models, limited to the population of caregivers to patient participants from communities of concern. | Measured at the 3-month and 6-month study visits | |
Other | Assessment of Social Support Effectiveness Questionnaire (SSE-Q): among members of communities of concern (low socioeconomic status, Hispanic ethnicity, or Black/Asian/Other race). | Investigators will assess Social Support Effectiveness Questionnaire (SSE-Q) within the cohort of patient participants who are members of communities of concern. In an intention-to-treat assessment, investigators will compare mean change in patient's SSE-Q from 3 months to 6 months between patients whose caregivers were randomized to the intervention versus control using linear mixed models among patient participants who are members of communities of concern. The SSE-Q is a validated 26-item measure of social support with higher scores indicating more effective support. The overall scale ranges from 0 to 80 and each subscale can range from 0 to 20. There are 4 subscales: task support, informational support, emotional support and negative effects of support. The first 15 questions are rated on a 5-point Likert scale and the last questions have yes/no responses. The SSE-Q has not previously been utilized in the CF population. | Measured at the 3-month and 6-month study visits | |
Other | Assessment of Brief Assessment Scale for Caregivers (BASC): by caregiver gender | Investigators will assess Brief Assessment Scale for Caregivers (BASC) by caregiver gender, as data in other diseases suggest that women may experience higher levels of caregiver burden and may have a different response to the intervention. In an intention-to-treat assessment, investigators will evaluate the difference in caregivers' mean change in BASC in the intervention versus control arms of the study from the 3-month to the 6-month study visit using linear mixed models, stratified by caregiver gender. The BASC is a validated 14-item measure of caregiver burden and includes subscales that reflect positive personal impact (PPI) and negative personal impact (NPI) of caregiving responsibilities. The BASC includes a 4-point Likert scale (scored 0 to 3), with higher scores indicating more burden, and the overall BASC score is an average of the 14 item scores. | Measured at the 3-month and 6-month study visits | |
Primary | Brief Assessment Scale for Caregivers (BASC) | The co-primary endpoint is an intention-to-treat assessment of the difference in caregivers' mean change in Brief Assessment Scale for Caregivers (BASC) in the intervention versus control arms of the study from the 3-month to the 6-month study visit using linear mixed models. The BASC is a validated 14-item measure of caregiver burden and includes subscales that reflect positive personal impact (PPI) and negative personal impact (NPI) of caregiving responsibilities. The BASC includes a 4-point Likert scale (scored 0 to 3), with higher scores indicating more burden, and the overall BASC score is an average of the 14 item scores. The BASC has high internal consistency in caregivers for adults with CF (Cronbach's alpha 0.88 for overall BASC and 0.82 for PPI). | Measured at the 3-month and 6-month study visits | |
Primary | Social Support Effectiveness Questionnaire (SSE-Q) | A co-primary endpoint is an intention-to-treat assessment of change in patient's Social Support Effectiveness Questionnaire (SSE-Q) from 3 months to 6 months and investigators will compare mean change between patients whose caregivers were randomized to the intervention versus control using linear mixed models. The SSE-Q is a validated 26-item measure of social support with higher scores indicating more effective support. The overall scale ranges from 0 to 80 and each subscale can range from 0 to 20. There are 4 subscales: task support, informational support, emotional support and negative effects of support. The first 15 questions are rated on a 5-point Likert scale and the last questions have yes/no responses. The SSE-Q has not previously been utilized in the CF population. | Measured at the 3-month and 6-month study visits | |
Secondary | Confidence-weighted true false knowledge about lung transplant (14-question investigator-designed survey) | Difference in mean change in confidence-weighted true false (CTF) knowledge score will be measured in the intervention versus control arms of the study using linear mixed models. CTF scoring adds points for certainty in correct responses and deducts points for certainty in incorrect responses. Participants receive +2 points when "sure" about a correct response, +1 if unsure about a correct response, -1 if unsure about an incorrect response and -2 if sure about an incorrect response. For a 14-item knowledge test, the maximum score is +28 and the minimum score is -28, with higher scores indicating more knowledge about lung transplant. | Measured at the 3-month and 6-month study visits | |
Secondary | Likert-scale rating of preparedness to discuss lung transplant with their loved one (0-4) | Likert rating of preparedness to discuss lung transplant with their loved one will be measured at each study visit (0=Don't know, 1= Not at all prepared, 2 = A little prepared, 3 = Moderately prepared, 4 = Very prepared). The intention-to-treat analysis will compare mean change in Likert-scale rating between the intervention and control arms of the study using linear mixed models. | Measured at the 3-month and 6-month study visits | |
Secondary | Patient Health Questionnaire (PHQ-8) | PHQ-8 is a scale that measures symptoms of depression in the prior 2 weeks on a 0-24 scale, with higher scores indicating worsening depression and a score of 10 or higher consistent with a diagnosis of depression. The intention-to-treat analysis will compare mean change in PHQ-8 between the intervention and control arms of the study using linear mixed models. Investigators will also determine the proportion with new PHQ-8 score greater than or equal to 10 in each study arm. | Measured at the 3-month and 6-month study visits | |
Secondary | Generalized Anxiety Disorder 7-item (GAD-7) Scale | GAD-7 is a scale that measures symptoms of anxiety in the prior 2 weeks on a 0-21 scale, with higher scores indicating worsening anxiety and a score of 10 or higher consistent with a diagnosis of generalized anxiety disorder. The intention-to-treat analysis will compare mean change in GAD-7 between the intervention and control arms of the study using linear mixed models. Investigators will also determine the proportion with new GAD-7 score greater than or equal to 10 in each study arm. | Measured at the 3-month and 6-month study visits |
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